Voretigene neparvovec

Voretigene neparvovec ( Luxturna ) is a novel gene therapy for the treatment of Leber’s congenital amaurosis . It was developed by Spark Therapeutics and Children’s Hospital of Philadelphia . [1] [2] It is the first in vivo gene therapy approved by the FDA. [3]

Leber’s congenital amaurosis, or biallelic RPE65- mediated inherited retinal disease, is an inherited progressive progressive blindness. Voretigene is the first treatment for this condition. [4]The gene therapy is not a cure for the condition, but rather improves vision in those treated. [5] It is given as an subretinal injection.

The price of the treatment has not been announced, but it is expected to cost around $ 1 million. [3] [6]

Development

Voretigene neparvovec is an AAV2 vector containing human RPE65 cDNA with a modified Kozak sequence . The virus is grown in HEK 293 cells and purified for administration. [7]

It was granted orphan drug status for Leber congenital amaurosis and retinitis pigmentosa . [8] [ better source needed ] A biologics license application was submitted to the FDA in July 2017 with Priority Review . [4] Phase III clinical trial results were published in August 2017. [9] On October 12, 2017, a key advisory panel to the Food and Drug Administration (FDA) , composed of 16 experts, unanimously recommended approval of the treatment. [10] The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received apediatric disease priority review voucher . [11]